JP2013518899A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2013518899A5 JP2013518899A5 JP2012552100A JP2012552100A JP2013518899A5 JP 2013518899 A5 JP2013518899 A5 JP 2013518899A5 JP 2012552100 A JP2012552100 A JP 2012552100A JP 2012552100 A JP2012552100 A JP 2012552100A JP 2013518899 A5 JP2013518899 A5 JP 2013518899A5
- Authority
- JP
- Japan
- Prior art keywords
- genome
- aav
- vector
- dystrophin
- composition according
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- GXJABQQUPOEUTA-RDJZCZTQSA-N Bortezomib Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)B(O)O)NC(=O)C=1N=CC=NC=1)C1=CC=CC=C1 GXJABQQUPOEUTA-RDJZCZTQSA-N 0.000 claims 12
- 229960001467 bortezomib Drugs 0.000 claims 12
- 239000008194 pharmaceutical composition Substances 0.000 claims 12
- 102000001690 Factor VIII Human genes 0.000 claims 10
- 108010054218 Factor VIII Proteins 0.000 claims 10
- 229960000301 Factor VIII Drugs 0.000 claims 10
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 8
- 229940099039 Velcade Drugs 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 102000001039 Dystrophin Human genes 0.000 claims 5
- 108010069091 Dystrophin Proteins 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 102000034448 gene-regulatory proteins Human genes 0.000 claims 4
- 108091006088 gene-regulatory proteins Proteins 0.000 claims 4
- 241000432074 Adeno-associated virus Species 0.000 claims 3
- 229920001405 Coding region Polymers 0.000 claims 3
- 210000004027 cells Anatomy 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 239000003937 drug carrier Substances 0.000 claims 2
- 239000000825 pharmaceutical preparation Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 102100000368 F8 Human genes 0.000 claims 1
- 101700070229 F8 Proteins 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 210000004185 Liver Anatomy 0.000 claims 1
- 208000003627 Muscular Dystrophy Diseases 0.000 claims 1
- 201000003883 cystic fibrosis Diseases 0.000 claims 1
- 201000003542 factor VIII deficiency Diseases 0.000 claims 1
- 210000000663 muscle cells Anatomy 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
Claims (19)
（ｂ）ボルテゾミブ（ベルケイド（登録商標））と
を含む組成物。 (A) an adeno-associated virus (AAV) vector comprising a heterologous nucleic acid encoding a protein selected from the group consisting of Factor VIII (FVIII), dystrophin, mini-dystrophin and transmembrane regulator protein (CFTR) , A vector in which the genome of the AAV vector is excessive compared to the wild type AAV genome;
(B) A composition comprising bortezomib (Velcade (registered trademark)) .
（ａ）前記核酸を含むアデノ随伴ウイルス（ＡＡＶ）ベクターであって、ＡＡＶベクターのゲノムが、野生型ＡＡＶゲノムに比して過大であるベクターと、
（ｂ）ボルテゾミブ（ベルケイド（登録商標））と
を薬学的に許容される担体中に含む医薬製剤。 A pharmaceutical formulation for delivering to a cell a nucleic acid encoding a protein selected from the group consisting of Factor VIII (FVIII), dystrophin, mini-dystrophin and transmembrane regulator protein (CFTR) ,
(A) an adeno-associated virus (AAV) vector comprising the nucleic acid, wherein the AAV vector genome is larger than the wild-type AAV genome;
(B) Bortezomib (Velcade (registered trademark)) and
Including pharmaceutical preparations in a pharmaceutically acceptable carrier a.
（ｂ）ボルテゾミブ（ベルケイド（登録商標））と
を含むキット。 (A) an adeno-associated virus (AAV) vector comprising a heterologous nucleic acid encoding a protein selected from the group consisting of Factor VIII (FVIII), dystrophin, mini-dystrophin and transmembrane regulator protein (CFTR) , A vector in which the genome of the AAV vector is excessive compared to the wild type AAV genome;
(B) A kit containing bortezomib (Velcade (registered trademark)) .
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US30199810P | 2010-02-05 | 2010-02-05 | |
| US61/301,998 | 2010-02-05 | ||
| PCT/US2011/023715 WO2011097456A2 (en) | 2010-02-05 | 2011-02-04 | Compositions and methods for enhanced parvovirus transduction |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013518899A JP2013518899A (en) | 2013-05-23 |
| JP2013518899A5 true JP2013518899A5 (en) | 2015-02-26 |
| JP6141021B2 JP6141021B2 (en) | 2017-06-07 |
Family
ID=44356082
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012552100A Active JP6141021B2 (en) | 2010-02-05 | 2011-02-04 | Compositions and methods for enhancing parvovirus transduction |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US9169492B2 (en) |
| EP (1) | EP2531604B1 (en) |
| JP (1) | JP6141021B2 (en) |
| CA (1) | CA2788682C (en) |
| ES (1) | ES2628889T3 (en) |
| WO (1) | WO2011097456A2 (en) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2534172A2 (en) * | 2010-02-12 | 2012-12-19 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Compositions and methods for preventing or treating a human parvovirus infection |
| JP6516725B2 (en) * | 2013-04-08 | 2019-05-22 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | Chimeric Adeno-Associated Virus / Bocavirus Parvovirus Vector |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (en) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Aadc polynucleotides for treating parkinson's disease |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JP6863891B2 (en) | 2014-11-14 | 2021-04-21 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | Regulatory polynucleotide |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| ES2900973T3 (en) * | 2015-01-07 | 2022-03-21 | UNIV AUTòNOMA DE BARCELONA | Single vector genetic construct comprising insulin and glucokinase genes |
| EP3253786A4 (en) * | 2015-02-06 | 2018-10-17 | The University of North Carolina at Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
| DK3402530T3 (en) * | 2016-01-13 | 2020-11-23 | Genzyme Corp | RECOMBINANT AAV VECTORS EXPRESSING BONE PROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, WHICH ARE USABLE FOR THE TREATMENT OF OSTEOARTHRITIS AND RELATED JOINT CONDITIONS OF PATTEDY |
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US11684679B2 (en) | 2016-03-07 | 2023-06-27 | University Of Iowa Research Foundation | AAV-mediated expression using a synthetic promoter and enhancer |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| RU2018142273A (en) * | 2016-05-03 | 2020-06-03 | Чжо-хуа Пань | METHOD OF IMPROVING VIRUS-MEDIATED DELIVERY OF GENES TO THE EYE USING PROTEASOM INHIBITORS |
| IL302748A (en) | 2016-05-18 | 2023-07-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| BR112018073472A2 (en) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics Inc | Huntington's disease treatment compositions and methods |
| EP3500696A4 (en) | 2016-08-16 | 2020-04-08 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| WO2018039333A1 (en) | 2016-08-23 | 2018-03-01 | Bluebird Bio, Inc. | Tim3 homing endonuclease variants, compositions, and methods of use |
| CA3035522A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| EP3510157B1 (en) | 2016-09-08 | 2023-05-31 | 2seventy bio, Inc. | Pd-1 homing endonuclease variants, compositions, and methods of use |
| US11530395B2 (en) | 2016-10-17 | 2022-12-20 | 2Seventy Bio, Inc. | TGFBetaR2 endonuclease variants, compositions, and methods of use |
| IL266628B1 (en) | 2016-11-17 | 2024-02-01 | Bluebird Bio Inc | TGFBeta SIGNAL CONVERTOR |
| US11142775B2 (en) | 2017-01-13 | 2021-10-12 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| US10436089B2 (en) * | 2017-01-18 | 2019-10-08 | Cummins Emission Solutions Inc. | Radio frequency sensor in an exhaust aftertreatment system |
| CN110446781A (en) | 2017-02-15 | 2019-11-12 | 蓝鸟生物公司 | Donor recovery template multiple gene group editor |
| AU2018261790A1 (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| CN111108198A (en) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | Compositions and methods for treating huntington's disease |
| CA3064014A1 (en) | 2017-05-25 | 2018-11-29 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| JOP20190269A1 (en) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| US11497576B2 (en) | 2017-07-17 | 2022-11-15 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| JP7221275B2 (en) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Compositions and methods for delivering AAV |
| US11779654B2 (en) | 2017-10-04 | 2023-10-10 | 2Seventy Bio, Inc. | PCSK9 endonuclease variants, compositions, and methods of use |
| WO2019079240A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| WO2019200013A1 (en) * | 2018-04-11 | 2019-10-17 | Cancer Targeting Systems, Inc. | Therapeutic constructs for treating cancer |
| JP2021527409A (en) | 2018-06-14 | 2021-10-14 | ブルーバード バイオ， インコーポレイテッド | CD79A chimeric antigen receptor |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| MX2021012682A (en) * | 2019-04-15 | 2022-03-25 | Spirovant Sciences Inc | Methods and compositions for transgene expression. |
| JP2022550435A (en) * | 2019-10-04 | 2022-12-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | Methods for improved therapeutic use of recombinant AAV |
| TW202204383A (en) | 2020-04-10 | 2022-02-01 | 美商碩拿生物科學有限責任公司 | Compositions and methods for the treatment of protein aggregation disorders |
| WO2021222168A2 (en) | 2020-04-28 | 2021-11-04 | Sola Biosciences Llc | Compositions and methods for the treatment of tdp-43 proteinopathies |
| WO2021248038A1 (en) | 2020-06-05 | 2021-12-09 | Sola Biosciences Llc | Compositions and methods for the treatment of synucleinopathies |
| US20230374542A1 (en) | 2020-10-07 | 2023-11-23 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21) |
| CA3195071A1 (en) * | 2020-10-08 | 2022-04-14 | Inozyme Pharma, Inc. | Liver specific production of enpp1 or enpp3 |
| EP4232462A1 (en) | 2020-10-26 | 2023-08-30 | SOLA Biosciences LLC | Compositions and methods for the treatment of alzheimer's disease |
| AU2022256479A1 (en) | 2021-04-16 | 2023-11-30 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| WO2023288267A1 (en) | 2021-07-14 | 2023-01-19 | 2Seventy Bio, Inc. | Engineered t cell receptors fused to binding domains from antibodies |
| WO2023060221A2 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of proteopathies |
| CA3234720A1 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of p53-mediated cancers |
| WO2023196997A2 (en) | 2022-04-08 | 2023-10-12 | 2Seventy Bio, Inc. | Multipartite receptor and signaling complexes |
| WO2023230512A1 (en) | 2022-05-26 | 2023-11-30 | 2Seventy Bio, Inc. | Compositions for maintaining lentiviral vector and uses thereof |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
| JP4969002B2 (en) * | 1999-06-08 | 2012-07-04 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | Compounds and methods for increasing rAAV transduction |
| US7241447B1 (en) | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| CA2520028A1 (en) | 2003-03-31 | 2004-10-21 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| WO2007092944A2 (en) * | 2006-02-08 | 2007-08-16 | Introgen Therapeutics, Inc. | Compositions and methods involving gene therapy and proteasome modulation |
| EP2037892B1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| CN101711164B (en) | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
| WO2008127707A1 (en) * | 2007-04-13 | 2008-10-23 | Dana Farber Cancer Institute, Inc. | Receptor tyrosine kinase profiling |
-
2011
- 2011-02-04 WO PCT/US2011/023715 patent/WO2011097456A2/en active Application Filing
- 2011-02-04 EP EP11740407.9A patent/EP2531604B1/en active Active
- 2011-02-04 JP JP2012552100A patent/JP6141021B2/en active Active
- 2011-02-04 CA CA2788682A patent/CA2788682C/en active Active
- 2011-02-04 ES ES11740407.9T patent/ES2628889T3/en active Active
- 2011-02-04 US US13/575,975 patent/US9169492B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2013518899A5 (en) | ||
| Ling et al. | Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo | |
| JP2020513814A5 (en) | ||
| JP2018108113A5 (en) | ||
| Ruff et al. | Precision templating with DNA of a virus-like particle with peptide nanostructures | |
| Kotchey et al. | A potential role of distinctively delayed blood clearance of recombinant adeno-associated virus serotype 9 in robust cardiac transduction | |
| JP2011101646A5 (en) | ||
| WO2018192982A3 (en) | Methods for adeno-associated viral vector production | |
| JP2017536116A5 (en) | ||
| WO2015196179A1 (en) | Methods of packaging multiple adeno-associated virus vectors | |
| JP2002538770A5 (en) | ||
| MX2020000221A (en) | Non human great apes adenovirus nucleic acid- and amino acid-sequences, vectors containing same, and uses thereof. | |
| RU2020108189A (en) | COMPOSITIONS AND METHODS OF TREATMENT OF LATERAL AMYOTROPHIC SCLEROSIS (ALS) | |
| JP2019513393A5 (en) | ||
| JP2016538885A5 (en) | ||
| JP2016520290A5 (en) | ||
| WO2016081927A2 (en) | Genome-modified recombinant adeno-associated virus vectors | |
| WO2015081101A8 (en) | Adeno-associated virus vectors for treatment of glycogen storage disease | |
| JP2013517798A5 (en) | ||
| JP2008541759A5 (en) | ||
| JP2015513897A5 (en) | ||
| Okada et al. | Current challenges and future directions in recombinant AAV-mediated gene therapy of Duchenne muscular dystrophy | |
| JP2012519657A5 (en) | ||
| RU2015135890A (en) | VACCINE COMPOSITION | |
| Chaikeeratisak et al. | The phage nucleus and PhuZ spindle: Defining features of the subcellular organization and speciation of nucleus-forming jumbo phages |